B-cell-delivered gene therapy induces functional T regulatory cells and leads to a loss of antigen-specific effector cells

Mol Ther. 2010 Aug;18(8):1527-35. doi: 10.1038/mt.2010.95. Epub 2010 May 18.

Abstract

Previous reports have shown that B-cell-mediated gene therapy can induce tolerance in several animal models for autoimmune diseases and inhibitory antibody formation in hemophilia A mice. We know from our previous work that the induction of tolerance following B-cell therapy is dependent upon CD25(+) regulatory T cells (Tregs). To extend these studies and identify the effects of this gene therapy protocol on the target CD4 T cells, we have adapted in vitro suppression assays using Tregs isolated from treated and control mice. Using carboxyfluorescein succinimidyl ester (CFSE) dilution as a measure of T-cell responsiveness to FVIII, we show that CD25(+) Tregs from treated mice are more suppressive than those from control animals. To monitor the induction of antigen-specific Tregs, we repeated these studies in ovalbumin (OVA) peptide-specific DO11.10 T-cell receptor (TCR) transgenic mice. Tregs from DO11.10 mice treated with a tolerogenic OVA-Ig construct are better than polyclonal Tregs at suppressing the proliferation of responder cells stimulated with OVA peptide 323-339 (pOVA). Furthermore, we show that following B-cell therapy, there is an increase in antigen-specific FoxP3(+) Tregs, and there is also a distinct decrease in antigen-specific CD4(+) effector T cells. These changes in the lymphocyte population shift the balance away from effector function toward a tolerogenic phenotype.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CD4-Positive T-Lymphocytes / metabolism
  • Cells, Cultured
  • Disease Models, Animal
  • Genetic Therapy / methods*
  • Hemophilia A / immunology
  • Hemophilia A / therapy
  • Interleukin-2 Receptor alpha Subunit / metabolism
  • Mice
  • Mice, Inbred BALB C
  • Mice, Mutant Strains
  • T-Lymphocytes, Regulatory / immunology*
  • T-Lymphocytes, Regulatory / metabolism

Substances

  • Interleukin-2 Receptor alpha Subunit